National Database on Primary Sclerosing Cholangitis (PSC)

Sponsor

University of Milano Bicocca (Other)

Overall Status

Recruiting

CT.gov ID

NCT05618145

Collaborator

(none)

6,000

Enrollment

Locations

120

Anticipated Duration (Months)

95.2

Patients Per Site

0.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Primary sclerosing cholangitis (PSC) a rare, chronic fibroinflammatory disease of the liver.

No data about the disease epidemiology exist in Italy. Therefore this study aims to develop a national PSC patient database linked to a biological sample storage.

Condition or Disease	Intervention/Treatment	Phase
Primary Sclerosing Cholangitis	Other: Clinical information

Detailed Description

Primary sclerosing cholangitis is a chronic fibroinflammatory disease of the liver characterized by chronic inflammation and sclerosis of the intrahepatic and/or extrahepatic bile ducts, and a risk for progression to liver failure and development of colorectal and hepatobiliary cancer 1. Both children and adults are affected. Patients with PSC have a diminished life expectancy with a median survival of 17 years after diagnosis. PSC is the leading indication for transplantation in some European countries. Epidemiological studies have found the highest prevalence rates of PSC in Northern European countries and North America (United States and Canada) ranging between 3.85 to 16.2 per 100,000 persons. In Italy, the estimates is of 0.8 per 100,000 persons but these figures underestimate the real disease burden 2.

Liver transplantation is currently the only life-extending accepted therapy for patients with end-stage liver disease (ESLD) secondary to PSC, and patients with PSC complicated by CCA who meet specific criteria. PSC recurs in the transplanted liver in up to 40% of patients. Taken together and combined with patients' debilitating quality of life issues, these data highlight the considerable disease burden and clinical impact of PSC on patients' outcomes. There is a strong, yet poorly understood, relationship between PSC and IBD; nearly 70%-80% of PSC patients have IBD, mainly ulcerative colitis.

Despite the high mortality associated with PSC and the efforts to optimize its management, there is no medical therapy proven to halt the progression of PSC or prevent its serious complications.

There have been no epidemiologic studies in PSC carried out in Italy, with the exception of the report of prevalence from the National registry of rare disease which provided an underestimated prevalence of 500 patients across the country. However, this study is limited by the nature of the registry which has administrative purposes and has a high rate of underreported cases.

The aim of the study is to implement a nationwide data collection on this rare disease to describe incidence and prevalence of PSC in Italy; identify and define distinct phenotypes and sub-phenotypes of PSC patients; identify factors influencing the progression of PSC and affecting mortality; assess safety and long-term efficacy of novel therapies.

The investigators will recruit patients and organise the collection of important clinical information and laboratory investigation, together with biological samples. Data will be collected in the form of electronic Case Report Forms (REDCap cloud) that will be completed by clinicians at baseline and thereafter on an annual basis. The clinical information will allow us to identify patients' clinical profiles. The biological samples will allow to understand key aspects of people's make up.

Study Design

Study Type:

Observational

Anticipated Enrollment :

6000 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Multicenter, Nationwide, Observational, Prospective Study Based on the Development of a Primary Sclerosing Cholangitis Patient's Database Linked to a Biological Sample Storage

Actual Study Start Date :

Oct 21, 2022

Anticipated Primary Completion Date :

Oct 21, 2032

Anticipated Study Completion Date :

Oct 21, 2032

Arms and Interventions

Arm	Intervention/Treatment
PSC population residing in Italy All PSC patients living in Italy and aged at least 17 years can be included in the database.	Other: Clinical information The investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples.

Arm

Intervention/Treatment

PSC population residing in Italy

All PSC patients living in Italy and aged at least 17 years can be included in the database.

Other: Clinical information

The investigators will recruit PSC patients and collect important clinical information and laboratory investigation, together with biological samples.

Outcome Measures

Primary Outcome Measures

Identify and define distinct phenotypes and sub-phenotypes of PSC patients at higher risk of disease progression. [Overall duration of the study (10 years)]
Identify and define distinct phenotypes and sub-phenotypes of PSC patients at higher risk of disease progression.

Secondary Outcome Measures

Identification of factors associated with response to therapies; [Overall duration of the study (10 years)]
Identification of factors associated with response to therapies;
Identification of biomarkers influencing the progression of PSC and affecting mortality [Overall duration of the study (10 years)]
Identification of biomarkers influencing the progression of PSC and affecting mortality
Assessment of safety and long-term efficacy of novel therapies [Overall duration of the study (10 years)]
Assessment of safety and long-term efficacy of novel therapies

Eligibility Criteria

Criteria

Ages Eligible for Study:

17 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Willing and able to give informed consent prior to any study specific procedure being performed;
Diagnosis of PSC according to the most recent published guidelines (EASL);

Exclusion Criteria:

Subject unwilling to participate at the study

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Ospedali riuniti di Ancona	Ancona	Italy
2	Policlinico di Bari	Bari	Italy
3	ASST Bergamo ovest	Bergamo	Italy
4	ASST Papa Giovanni XXIII	Bergamo	Italy
5	Ospedale Sant'Orsola Malpighi	Bologna	Italy
6	Policlinico S. Orsola Malpighi	Bologna	Italy
7	Azienda sanitaria dell'Alto Adige	Bolzano	Italy
8	Spedali civili	Brescia	Italy
9	Unità epatologica ed ecografia internistica a valenza dipartimentale, Fondazione Ospedaliera Poliambulanza	Brescia	Italy
10	Policlinico universitario Monserrato	Cagliari	Italy
11	Presidio Ospedaliero San Michele ARNAS G. Brotzu	Cagliari	Italy
12	Osp. "S de Bellis" - IRCCS	Castellana Grotte	Italy
13	UO epatologia Ospedale Garibaldi	Catania	Italy
14	Azienda Ospedaliera Universitaria Mater Domini	Catanzaro	Italy
15	Azienda Ospedaliera Universitaria- Mater Domini	Catanzaro	Italy
16	Ospedale Valduce	Como	Italy
17	Unità operativa complessa di medicina interna- Azienda ospedaliera di Cosenza	Cosenza	Italy
18	ASST di Cremona	Cremona	Italy
19	Ospedale santa croce	Cuneo	Italy
20	Presidio Ospedaliero di Faenza (RA)	Faenza	Italy
21	Azienda Ospedaliero-Universitaria di Ferrara	Ferrara	Italy
22	Azienda ospedaliera universitaria careggi Firenze	Firenze	Italy
23	Azienda ospedaliera universitaria Careggi	Firenze	Italy
24	Centro C.U.R.E. Centro Universitario per la ricerca e la Cura delle Malattie Epatiche	Foggia	Italy
25	Ospedale Casa Sollievo della Sofferenza	Foggia	Italy
26	ASST Valle Olona	Gallarate	Italy
27	Policlinico San Martino	Genova	Italy
28	Presìdio Ospedaliero "Giovanni Paolo II"	Lamezia Terme	Italy
29	ASST Lecco	Lecco	Italy
30	UO di epatologia clinica e biomolecolare. Università degli studi di Messina	Messina	Italy
31	Az. Ospedaliera San Paolo- Polo universitario	Milano	Italy
32	Istituto scientifico universitario San Raffaele	Milano	Italy
33	Ospedale Niguarda Ca' Granda	Milano	Italy
34	Ospedale San Giuseppe	Milano	Italy
35	Policlinico di Milano	Milano	Italy
36	Dipartimento Ospedaliero di Medicina Interna	Modena	Italy
37	ASST Monza	Monza	Italy	20900
38	Fondazione Evangelica Villa Betania, Ospedale generale di Zona	Napoli	Italy
39	Ospedale policlinico Federico II di Napoli	Napoli	Italy
40	Università della Campania Luigi Vanvitelli	Napoli	Italy
41	Ospedale maggiore della carità	Novara	Italy
42	Azienda Ospedaliera di Padova	Padova	Italy
43	UO di Gastroenterologia Azienda ospedaliera di Padova	Padova	Italy
44	Policlinico Paolo Giaccone	Palermo	Italy
45	Azienda ospedaliero universitaria di Parma	Parma	Italy
46	Istituto Clinico Città di Pavia-Gruppo Sandonato	Pavia	Italy
47	Azienda ospedaliera di Perugia	Perugia	Italy
48	Day Hospital Internistico e ambulatorio di Epatologia, ASL Pescara	Pescara	Italy
49	Aou pisana Cisanello	Pisa	Italy
50	Ospedale di Pordenone AS FO	Pordenone	Italy
51	Ospedale San Carlo	Potenza	Italy
52	Azienda unità sanitaria locale della Romagna	Rimini	Italy
53	Ospedale A. Gemelli	Roma	Italy
54	Policlinico Umberto I	Roma	Italy
55	Policlinico universitario campus bio-medico	Roma	Italy
56	Universita' di Roma Tor Vergata	Roma	Italy
57	Istituto clinico Humanitas	Rozzano	Italy
58	Università degli studi di Salerno	Salerno	Italy
59	Azienda Ospedaliero-Universitaria Molinette	Torino	Italy
60	Azienda provinciale per i servizi sanitari	Trento	Italy
61	Ospedale di Cattinara, ASU GI	Trieste	Italy
62	ASU FC	Udine	Italy
63	ASST Sette Laghi	Varese	Italy

Sponsors and Collaborators

University of Milano Bicocca

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

University of Milano Bicocca

ClinicalTrials.gov Identifier:

NCT05618145

Other Study ID Numbers:

PSC_Database

First Posted:

Nov 16, 2022

Last Update Posted:

Nov 16, 2022

Last Verified:

Nov 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by University of Milano Bicocca

Primary Sclerosing Cholangitis

Disease phenotypes

National Database

Additional relevant MeSH terms:

Cholangitis

Cholangitis, Sclerosing

Study Results

No Results Posted as of Nov 16, 2022